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Spinal Muscular Atrophy (SMA)

Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy (SMA) is a genetic motor neuron disease.

SMN1 protein is critical for the maintenance of specialised nerve cells called motor neurons. Motor neurons are neurotransmitters that carry signals from the brain and spinal cord to the muscles in the rest of the body. They tell muscles when to tense and when to relax, the mechanism by which movement becomes possible. Spinal muscular atrophy is caused by a mutation that leads to the loss of SMN1 protein and its associated motor neurons. This loss inhibits the ability of the brain to ‘move’ the rest of the body. When muscles remain inactive for prolonged periods of time, they atrophy – which is to get smaller - eventually impairing swallowing and breathing.

How Our Products Can Treat Spinal Muscular Atrophy

To date, there is no cure for spinal muscular atrophy. Instead, what therapy can do is treat the symptoms of the disease with the most success.

We use products derived from human baby umbilical cord tissues in all our therapy programs - Wharton's Jelly MSCs (wjMSCs). They are special because they can:

  • prevent cells from dying (apoptosis) [1,2,3,4,5].
  • encourage existing cells to self-repair (autocrine effect) [6,7,8].
  • transform into neurons and glial cells [5,9,10,11,12,13].
  • promote the formation of nerve cell axons (axogenesis) [14,15].
  • prevent rejection by regulating the immune system (immunomodulation) [1,2,16,17,18,19,20,21,22].
  • reduce inflammation [2,4,9,14,21,22,23].
  • communicate with and alter nearby cells (paracrine effect) [3,6,24].
  • incapable of forming tumours [1,18,19,25].
  • release neuroprotective factors [3,26].
  • encourage existing cells to adapt (neuroplasticity) [27,28,29].

The human brain is strictly regulated by the blood-brain barrier (BBB). This is a semipermeable endothelial membrane that separates blood from cerebrospinal fluid. It is a highly selective mechanism that allows only a select few types of cells, particles and molecules enter the brain. In this respect, wjMSCs are known to be able to:

  • cross the endothelial brain barrier [30,31,32,33].
  • migrate to sites of injury (chemotaxis) [1,2].

 

Treating Spinal Muscular Atrophy

In the treatment of spinal muscular atrophy, the aim is to improve quality of life and to delay the progression of the disease by preventing additional damage. Human clinical trials for treating spinal muscular atrophy are in their infancy. To date there are three registered trials with no results published [34,35,36].

A Chinese study conducted on a child with SMA with umbilical cord blood MSCs in 2010 showed that levels of muscle enzymes decreased and Functional Independence Measure (FIM) scores increased by 25 points. Electromyography (EMG) results showed that motor units (with re-contraction) increased and that motor function was improved. At six months post-transplantation, the child exhibited significantly improved lower extremity muscle strength and self-care ability. During the 10-month follow-up, the child had experienced no adverse effects [37].

In 2012, the Bone Marrow Transplantation Department of Trieste, Italy treated four children suffering from SMA1 by means of intrathecal injections of bone marrow MSCs (bmMSCs). All patients experienced improved mobility after three weeks but no improvements were noted in intercostal muscles for breathing. Transplantations were repeated once a month for a period of three to eight months as the effect of each session did not last more than 30 days. The children experienced progressive improvements during and after the first three transplantations but their progress has since plateaued. However, subsequent therapy sessions seem to ensure that their newly acquired mobility is maintained [38].

In 2015, scientists from Rutgers New Jersey Medical School reported findings of three children with spinal muscular atrophy type 1 who were treated with bmMSCs in Italy. The first child experienced better head control, thigh abduction and adduction, legs and development of hand grip after five infusions. In addition, his general condition seemed improved, body weight increased, the face became more expressive, able to articulate vowel sounds and and he developed several minute periods of ventilator-free breathing ability.

The second patient also experienced good recovery of head control and faster lower limb movements as well as the ability to breathe unsupported when awake and to speak louder and longer. Her pediatrician documented constant and continuous improvement during the months of treatment with bmMSCs infusions.

With the third child, 60 degrees of thigh abduction-adduction and other functional benefits were noted by her neuropsychiatrist. Marked improvements were also documented by numerous professionals - her pediatricians noted improvement in speech audibility, diminished drooling, and less constipation; her physical therapist certified improved motor skills; and her speech therapist noted improvements in facial expression and voice volume.

However, all three children lost all these active movements and other noted benefits within months of discontinuation of therapy indicating that therapy has to be repeated at intervals of a few months for improvements to be maintained [39].

 

Cyrona’s Program

Achieving high standards in our work is of paramount importance to us. Depending on a patient’s needs, we combine our premium grade Passage 2 wjMSCs with physiotherapy, occupational therapy, speech and language therapy and/or rehabilitative medicine. 

Learn more about our Products and Programs.

 

Why Choose Cyrona?

  • Latest cellular research and technology.
  • Unique, tailored therapy outlines.
  • Products that meet international standards.
  • Microbiology & clinical team with extensive experience in advanced medicine.
  • Board-certified physicians geared towards patient safety.
  • Fact-based information from clinical studies and trials.
  • No outlandish promises of a one-stop-cure or false improvement rates.

 

Therapy Packages

All our therapy packages come inclusive of:

 

INTERNATIONAL PATIENTS

  • Premium grade Passage 2 wjMSCs.
  • Treatment by qualified specialist(s).
  • Certificate of Analysis (CoA).
  • Airport transfer.
  • Transportation to & from therapy session(s).
  • Accommodation.
  • Hospital room for therapy.

 

LOCAL PATIENTS

  • Premium grade Passage 2 wjMSCs.
  • Treatment by qualified specialist(s).
  • Certificate of Analysis (CoA).
  • Transportation to & from therapy session(s).
  • Hospital room for therapy.

 

How Do We Proceed

All our therapies are charged based on the number of wjMSCs and supplementary infusions required for the patient’s specific condition. As no two people are alike, our specialists review each patient’s medical reports before tailoring a therapy catered to addressing his or her individual needs.

You may chat with one of our Customer Care Representatives or send an e-mail detailing the patient’s condition to one of our Liaison Officers. It would expedite the process if you can provide us with:

  • Imaging results (MRI scan / CT scan / X-Ray).
  • Hematology reports (blood test).
  • Doctor’s assessment reports.
  • Pictures or videos of the patient (if relevant).

Upon getting in touch with us, a Liaison Officer evaluates and assigns the case to the specialist best equipped to treat the condition. A therapy, unique only to the patient, is drawn up and a price quoted accordingly.

Should you decide to proceed with therapy, our specialists require that all patients have Cancer Marker Screening performed in their country of residence before travelling to us for therapy. If the patient has had Cancer Marker Screening within the last 3 months, you may e-mail those results to us. In the event that the patient’s Cancer Marker Screening results are not satisfactory, our specialists will refuse to proceed with therapy. It is for this reason that we requests that patients have Cancer Marker Screening performed in their country of residence prior to travelling to us.

One week prior to arrival, a deposit payment is required in order to arrange accommodation and transportation.

Full payment is required to be made one-day prior to therapy commencement.

Post-treatment, our specialist will provide the patient with a post-treatment protocol as well as what to expect on his or her journey towards a better, and hopefully, healthier new life.

 

Kindly get in touch with us for the sources listed throughout this article.

 

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